Colchicine or Methotrexate, With Ursodiol, Are Effective After 20 Years in a Subset of Patients With Primary Biliary Cirrhosis

BACKGROUND & AIMS: The combination of ursodeoxycholic acid (UDCA), colchicine, and methotrexate (MTX) is effective therapy for a subset of patients with primary biliary cirrhosis (PBC) who do not respond to UDCA. However, the durability of the response is unclear. We investigated whether the response to combination therapy was durable. METHODS: We followed, for 10 additional years (range 9-13 years), 29 patients with PBC who had been treated with the combination of UDCA and MTX or UDCA and colchicine in a randomized controlled trial that began in 1988 and lasted 10 years. RESULTS: Of the 11 patients given MTX plus UDCA, 9 were still alive and well, whereas 2 died from causes unrelated to liver disease at the ages of 79 and 70. Of the 18 patients given the combination of colchicine and UDCA, 12 were alive and well 20 years after the trial ended. Three had progressive liver disease; 2 of these had liver transplantation and 1 died of pneumonia. Three died of unrelated causes at the ages of 73, 76, and 76 years, respectively. CONCLUSIONS: Treatment with the combination of UDCA and MTX or UDCA and colchicine led to sustained clinical remission in a subset of patients with PBC. The response to the combination of UDCA and MTX appeared to be more durable than to UDCA and colchicine.